Access to Therapies For All FSHD-Affected Individuals
Access to Therapies For All FSHD-Affected Individuals
The Issue
Imagine there is a treatment for your or your family member's muscle wasting disease, but you can't get access to it
Right now, children, older adults, and non-ambulatory individuals of all ages with Facioscapulohumeral Muscular Dystrophy (FSHD) are being left out of interventional trials—despite the surge of promising therapies in development. These exclusions deny the most vulnerable in our community the chance at treatment and a better future.
FSHD is a progressive genetic disorder that causes muscle wasting, leading to profound physical limitations and, in some cases, life-threatening respiratory and heart conditions.
There are promising treatments in development, and yet over 50% of FSHD patients are being shut out from access due to restrictive inclusion criteria to clinical trials. This is not just a bureaucratic oversight — it is a failure of compassion, and it is leaving thousands of individuals with FSHD behind unnecessarily. Those who do not fit within the narrow patient profile used in trials are left with limited to no access to treatments due to FDA labeling and/or insurance coverage for these treatments. This is simply due to the lack of data available to approve treatments for everyone.
It's critical to design trials that are inclusive and equitable for the entire FSHD community.
We urge regulators and drug developers to:
- Implement innovative trial designs, such as open-label arms, adaptive designs, expanded inclusion criteria
- Establish Expanded Access Programs for those most severely affected who don’t qualify for trials.
- Ensure the FDA grants broader labels for FSHD therapies, ensuring treatments are available to all patients—not just a select minority—while additional data is gathered with confirmatory trials.
By allowing broader access, we can gather real-world data on safety, efficacy, and proper dosing, ensuring insurance coverage and better treatment decisions for the entire FSHD community.
No one should be left behind. Empower yourself, friends, and family members living with FSHD. Other neuromuscular communities fought these battles and won. Now it’s our turn.
Please sign this petition showing your support in this initiative demanding that every FSHD patient—regardless of age or mobility—has the right to access life-changing treatments. Together, we can make sure no one is excluded from a future free of FSHD.
Regan-Udall Expanded Access Information
Access to Novel Medical Products Podcast
1,732
The Issue
Imagine there is a treatment for your or your family member's muscle wasting disease, but you can't get access to it
Right now, children, older adults, and non-ambulatory individuals of all ages with Facioscapulohumeral Muscular Dystrophy (FSHD) are being left out of interventional trials—despite the surge of promising therapies in development. These exclusions deny the most vulnerable in our community the chance at treatment and a better future.
FSHD is a progressive genetic disorder that causes muscle wasting, leading to profound physical limitations and, in some cases, life-threatening respiratory and heart conditions.
There are promising treatments in development, and yet over 50% of FSHD patients are being shut out from access due to restrictive inclusion criteria to clinical trials. This is not just a bureaucratic oversight — it is a failure of compassion, and it is leaving thousands of individuals with FSHD behind unnecessarily. Those who do not fit within the narrow patient profile used in trials are left with limited to no access to treatments due to FDA labeling and/or insurance coverage for these treatments. This is simply due to the lack of data available to approve treatments for everyone.
It's critical to design trials that are inclusive and equitable for the entire FSHD community.
We urge regulators and drug developers to:
- Implement innovative trial designs, such as open-label arms, adaptive designs, expanded inclusion criteria
- Establish Expanded Access Programs for those most severely affected who don’t qualify for trials.
- Ensure the FDA grants broader labels for FSHD therapies, ensuring treatments are available to all patients—not just a select minority—while additional data is gathered with confirmatory trials.
By allowing broader access, we can gather real-world data on safety, efficacy, and proper dosing, ensuring insurance coverage and better treatment decisions for the entire FSHD community.
No one should be left behind. Empower yourself, friends, and family members living with FSHD. Other neuromuscular communities fought these battles and won. Now it’s our turn.
Please sign this petition showing your support in this initiative demanding that every FSHD patient—regardless of age or mobility—has the right to access life-changing treatments. Together, we can make sure no one is excluded from a future free of FSHD.
Regan-Udall Expanded Access Information
Access to Novel Medical Products Podcast
1,732
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Petition created on February 24, 2025